Technology Breakthroughs of 2025
Saving one life at a time: Gene-editing in clinical use Baby KJ was born in August 2024 with an ultra-rare genetic condition – carbamoyl-phosphate synthetase (CPS1) deficiency. The CPS1 enzyme, in the liver, converts ammonia (from broken down proteins) to carbamoyl phosphate. Mutations in the CPS1 gene cause carbamoyl phosphate synthetase I deficiency. CPS1 deficiency affects 1 in 1,300,000 persons. Defects in […]